EP4013428A4 - Modifizierte oligomere verbindungen und verwendungen davon - Google Patents

Modifizierte oligomere verbindungen und verwendungen davon Download PDF

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Publication number
EP4013428A4
EP4013428A4 EP20853483.4A EP20853483A EP4013428A4 EP 4013428 A4 EP4013428 A4 EP 4013428A4 EP 20853483 A EP20853483 A EP 20853483A EP 4013428 A4 EP4013428 A4 EP 4013428A4
Authority
EP
European Patent Office
Prior art keywords
oligomeric compounds
modified oligomeric
modified
compounds
oligomeric
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
EP20853483.4A
Other languages
English (en)
French (fr)
Other versions
EP4013428A1 (de
Inventor
Punit P. Seth
Michael T. Migawa
Graeme C. FREESTONE
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Ionis Pharmaceuticals Inc
Original Assignee
Ionis Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of EP4013428A1 publication Critical patent/EP4013428A1/de
Publication of EP4013428A4 publication Critical patent/EP4013428A4/de
Pending legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
EP20853483.4A 2019-08-15 2020-08-14 Modifizierte oligomere verbindungen und verwendungen davon Pending EP4013428A4 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962887534P 2019-08-15 2019-08-15
PCT/US2020/046540 WO2021030763A1 (en) 2019-08-15 2020-08-14 Modified oligomeric compounds and uses thereof

Publications (2)

Publication Number Publication Date
EP4013428A1 EP4013428A1 (de) 2022-06-22
EP4013428A4 true EP4013428A4 (de) 2023-08-09

Family

ID=74571252

Family Applications (1)

Application Number Title Priority Date Filing Date
EP20853483.4A Pending EP4013428A4 (de) 2019-08-15 2020-08-14 Modifizierte oligomere verbindungen und verwendungen davon

Country Status (3)

Country Link
US (1) US20230159922A1 (de)
EP (1) EP4013428A4 (de)
WO (1) WO2021030763A1 (de)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN114555621B (zh) 2019-08-15 2025-10-28 Ionis制药公司 键修饰的寡聚化合物及其用途
WO2021076543A1 (en) 2019-10-14 2021-04-22 Aro Biotherapeutics Company Epcam binding fibronectin type iii domains
AU2022258584A1 (en) 2021-04-14 2023-10-12 Aro Biotherapeutics Company Fn3 domain-sirna conjugates and uses thereof
TW202304971A (zh) 2021-04-14 2023-02-01 美商亞羅生物治療公司 結合cd71之纖連蛋白iii型結構域
WO2023023550A1 (en) * 2021-08-18 2023-02-23 Ionis Pharmaceuticals, Inc. Linkage modified oligomeric compounds and uses thereof
CN114213366A (zh) * 2022-01-17 2022-03-22 江西师范大学 一种氟代糖内酯合成方法
CN114539337A (zh) * 2022-02-28 2022-05-27 梯尔希（南京）药物研发有限公司 一种索非布韦杂质的制备方法
CN116143848A (zh) * 2023-01-30 2023-05-23 河南省三生药业有限公司 阿兹夫定关键中间体1-乙酰氧基-2,3,5-三苯甲酰氧基-1-beta-D-呋喃核糖制备方法
WO2025006955A2 (en) * 2023-06-30 2025-01-02 Ionis Pharmaceuticals, Inc. Cd29 targeted oligonucleotides and uses thereof
WO2025240884A1 (en) * 2024-05-17 2025-11-20 Ionis Pharmaceuticals, Inc. Patterned modified oligonucleotides

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6316612B1 (en) * 1997-08-22 2001-11-13 Ribozyme Pharmaceuticals, Inc. Xylofuranosly-containing nucleoside phosphoramidites and polynucleotides
WO2009146556A1 (en) * 2008-06-05 2009-12-10 The Royal Institution For The Advancement Of Learning/Mcgill University Oligonucleotide duplexes comprising dna-like and rna-like nucleotides and uses thereof

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6025335A (en) * 1995-09-21 2000-02-15 Lipitek International, Inc. L-Nucleoside Dimer Compounds and therapeutic uses
WO2003039523A2 (en) * 2001-11-05 2003-05-15 Exiqon A/S OLIGONUCLEOTIDES MODIFIED WITH NOVEL α-L-RNA ANALOGUES
EP3861118A4 (de) * 2018-10-05 2023-11-15 Ionis Pharmaceuticals, Inc. Modifizierte oligomere verbindungen und verwendungen davon

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6316612B1 (en) * 1997-08-22 2001-11-13 Ribozyme Pharmaceuticals, Inc. Xylofuranosly-containing nucleoside phosphoramidites and polynucleotides
WO2009146556A1 (en) * 2008-06-05 2009-12-10 The Royal Institution For The Advancement Of Learning/Mcgill University Oligonucleotide duplexes comprising dna-like and rna-like nucleotides and uses thereof

Non-Patent Citations (13)

* Cited by examiner, † Cited by third party
Title
G. F. DELEAVEY ET AL: "Synergistic effects between analogs of DNA and RNA improve the potency of siRNA-mediated gene silencing", NUCLEIC ACIDS RESEARCH, vol. 38, no. 13, 22 April 2010 (2010-04-22), pages 4547 - 4557, XP055057743, ISSN: 0305-1048, DOI: 10.1093/nar/gkq181 *
GATTA ADITYA KIRAN ET AL: "Strategies for improving the specificity of siRNAs for enhanced therapeutic potential", EXPERT OPINION ON DRUG DISCOVERY, vol. 13, no. 8, 14 June 2018 (2018-06-14), London, GB, pages 709 - 725, XP093060375, ISSN: 1746-0441, DOI: 10.1080/17460441.2018.1480607 *
GHOSH SOUMADWIP ET AL: "Spontaneous Unzipping of Xylonucleic Acid Assisted by a Single-Walled Carbon Nanotube: A Computational Study", JOURNAL OF PHYSICAL CHEMISTRY PART B, vol. 120, no. 15, 5 April 2016 (2016-04-05), US, pages 3642 - 3652, XP093058648, ISSN: 1520-6106, DOI: 10.1021/acs.jpcb.6b02035 *
GOSSELIN GILLES ET AL: "Systematic synthesis and biological evaluation of .alpha.- and .beta.-D-xylofuranosyl nucleosides of the five naturally occurring bases in nucleic acids and related analogs", JOURNAL OF MEDICINAL CHEMISTRY, vol. 29, no. 2, February 1986 (1986-02-01), US, pages 203 - 213, XP093058730, ISSN: 0022-2623, Retrieved from the Internet <URL:https://pubs.acs.org/doi/pdf/10.1021/jm00152a007> DOI: 10.1021/jm00152a007 *
MAITI MOHITOSH ET AL: "Xylonucleic acid: synthesis, structure, and orthogonal pairing properties", NUCLEIC ACIDS RESEARCH, vol. 43, no. 15, 14 July 2015 (2015-07-14), GB, pages 7189 - 7200, XP093058725, ISSN: 0305-1048, Retrieved from the Internet <URL:https://watermark.silverchair.com/gkv719.pdf?token=AQECAHi208BE49Ooan9kkhW_Ercy7Dm3ZL_9Cf3qfKAc485ysgAAAtQwggLQBgkqhkiG9w0BBwagggLBMIICvQIBADCCArYGCSqGSIb3DQEHATAeBglghkgBZQMEAS4wEQQMY8JmohXGCIxxLI4MAgEQgIICh22sCeFcjeeXlUe6MuD3rdMpVzHOUZcrOID0NOLGu9GndMF1tIiWJP5yEwDJp-aQwEeppge0O64u0lIzNItetjju3djvF> DOI: 10.1093/nar/gkv719 *
MARK K. SCHLEGEL ET AL: "Chirality Dependent Potency Enhancement and Structural Impact of Glycol Nucleic Acid Modification on siRNA", JOURNAL OF THE AMERICAN CHEMICAL SOCIETY, vol. 139, no. 25, 19 June 2017 (2017-06-19), pages 8537 - 8546, XP055602525, ISSN: 0002-7863, DOI: 10.1021/jacs.7b02694 *
MOHITOSH MAITI ET AL: "Solution Structure and Conformational Dynamics of Deoxyxylonucleic Acids (dXNA): An Orthogonal Nucleic Acid Candidate", CHEMISTRY - A EUROPEAN JOURNAL, JOHN WILEY & SONS, INC, DE, vol. 18, no. 3, 16 December 2011 (2011-12-16), pages 869 - 879, XP071835737, ISSN: 0947-6539, DOI: 10.1002/CHEM.201102509 *
POOPEIKO N E ET AL: "Xylo-Configured Oligonucleotides (XNA, Xylo Nucleic Acid): Synthesis of Conformationally Restricted Derivatives and Hybridization Towards DNA and RNA Complements", BIOORGANIC & MEDICINAL CHEMISTRY LETTERS, ELSEVIER, AMSTERDAM NL, vol. 13, 4 June 2003 (2003-06-04), pages 2285 - 2290, XP003018114, ISSN: 0960-894X, DOI: 10.1016/S0960-894X(03)00441-4 *
POOPEIKO NICOLAI E. ET AL: "xylo -Configured Oligonucleotides (XNA, Xylo Nucleic Acids): Synthesis and Hybridization Studies", NUCLEOSIDES, NUCLEOTIDES & NUCLEIC ACIDS, vol. 22, no. 5-8, October 2003 (2003-10-01), US, pages 1147 - 1149, XP093058745, ISSN: 1525-7770, DOI: 10.1081/NCN-120022822 *
PRAKASH THAZHA P ET AL: "Synergistic effect of phosphorothioate, 5'-vinylphosphonate and GalNAc modifications for enhancing activity of synthetic siRNA", BIOORGANIC & MEDICINAL CHEMISTRY LETTERS, ELSEVIER, AMSTERDAM NL, vol. 26, no. 12, 27 April 2016 (2016-04-27), pages 2817 - 2820, XP029557115, ISSN: 0960-894X, DOI: 10.1016/J.BMCL.2016.04.063 *
RAVINDRA BABU B ET AL: "XNA (xylo Nucleic Acid): A Summary and New Derivatives", EUROPEAN JOURNAL OF ORGANIC CHEMISTRY, WILEY-VCH, DE, vol. 2005, no. 11, 25 May 2005 (2005-05-25), pages 2297 - 2321, XP072105834, ISSN: 1434-193X, DOI: 10.1002/EJOC.200500023 *
SCHOPPE ARNULF ET AL: "Xylose-DNA: Comparison of the Thermodynamic Stability of Oligo(2'-deoxyxylonucleotide) and Oligo(2'-deoxyribonucleotide) Duplexes", EUROPEAN JOURNAL OF BIOCHEMISTRY, vol. 239, no. 1, July 1996 (1996-07-01), pages 33 - 41, XP093058747, ISSN: 0014-2956, DOI: 10.1111/j.1432-1033.1996.0033u.x *
See also references of WO2021030763A1 *

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Publication number Publication date
EP4013428A1 (de) 2022-06-22
US20230159922A1 (en) 2023-05-25
WO2021030763A1 (en) 2021-02-18

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